WO2017162265A1 - Arn à réplication trans - Google Patents

Arn à réplication trans Download PDF

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Publication number
WO2017162265A1
WO2017162265A1 PCT/EP2016/056160 EP2016056160W WO2017162265A1 WO 2017162265 A1 WO2017162265 A1 WO 2017162265A1 EP 2016056160 W EP2016056160 W EP 2016056160W WO 2017162265 A1 WO2017162265 A1 WO 2017162265A1
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WO
WIPO (PCT)
Prior art keywords
rna
replicase
alphavirus
present
replicon
Prior art date
Application number
PCT/EP2016/056160
Other languages
English (en)
Inventor
Tim Beissert
Ugur Sahin
Mario Perkovic
Original Assignee
Biontech Rna Pharmaceuticals Gmbh
Tron - Translationale Onkologie An Der Universitätsmedizin Der Johannes Gutenberg-Universität Mainz Gemeinnützige Gmbh
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Biontech Rna Pharmaceuticals Gmbh, Tron - Translationale Onkologie An Der Universitätsmedizin Der Johannes Gutenberg-Universität Mainz Gemeinnützige Gmbh filed Critical Biontech Rna Pharmaceuticals Gmbh
Priority to PCT/EP2016/056160 priority Critical patent/WO2017162265A1/fr
Priority to DK17710268.8T priority patent/DK3433368T3/da
Priority to EP17710268.8A priority patent/EP3433368B1/fr
Priority to SI201730973T priority patent/SI3433368T1/sl
Priority to AU2017236240A priority patent/AU2017236240B2/en
Priority to CA3017285A priority patent/CA3017285A1/fr
Priority to BR112018068391A priority patent/BR112018068391A2/pt
Priority to HUE17710268A priority patent/HUE058889T2/hu
Priority to EP21200788.4A priority patent/EP3964584A1/fr
Priority to CN202211115420.5A priority patent/CN115386596A/zh
Priority to US16/086,127 priority patent/US20200299724A1/en
Priority to PL17710268T priority patent/PL3433368T3/pl
Priority to HRP20211715TT priority patent/HRP20211715T1/hr
Priority to KR1020187027526A priority patent/KR102441725B1/ko
Priority to PCT/EP2017/055813 priority patent/WO2017162461A1/fr
Priority to MA44479A priority patent/MA44479B1/fr
Priority to PT177102688T priority patent/PT3433368T/pt
Priority to MDE20190126T priority patent/MD3433368T2/ro
Priority to RS20211377A priority patent/RS62543B1/sr
Priority to JP2018549306A priority patent/JP7105193B2/ja
Priority to ES17710268T priority patent/ES2896927T3/es
Priority to IL261327A priority patent/IL261327B1/en
Priority to SG11201807378SA priority patent/SG11201807378SA/en
Priority to MX2018011384A priority patent/MX2018011384A/es
Priority to CN201780018575.8A priority patent/CN109328233B/zh
Priority to LTEPPCT/EP2017/055813T priority patent/LT3433368T/lt
Priority to RU2018131783A priority patent/RU2752580C2/ru
Publication of WO2017162265A1 publication Critical patent/WO2017162265A1/fr
Priority to ZA201805520A priority patent/ZA201805520B/en
Priority to ZA2019/05396A priority patent/ZA201905396B/en
Priority to JP2021160491A priority patent/JP7421256B2/ja
Priority to CY20211100949T priority patent/CY1124689T1/el

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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1131—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N2770/36111—Alphavirus, e.g. Sindbis virus, VEE, EEE, WEE, Semliki
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    • C12N2770/36111—Alphavirus, e.g. Sindbis virus, VEE, EEE, WEE, Semliki
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    • C12N2770/36011—Togaviridae
    • C12N2770/36111—Alphavirus, e.g. Sindbis virus, VEE, EEE, WEE, Semliki
    • C12N2770/36141—Use of virus, viral particle or viral elements as a vector
    • C12N2770/36143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
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Abstract

La présente invention concerne de manière générale des systèmes et des procédés aptes à produire des protéines de haut niveau. Tandis qu'un ou plusieurs élément(s) de la présente invention est/sont dérivé(s) d'un alphavirus, la présente invention ne nécessite pas de propagation de particules virales. En particulier, l'invention concerne un système comportant deux molécules d'ARN séparées, comprenant chacune une séquence nucléotidique dérivée d'un alphavirus: une molécule d'ARN comprend une construction d'ARN pour exprimer une réplicase d'alphavirus, et une molécule d'ARN comprend un réplicon d'ARN qui peut être répliqué par la réplicase en trans. La construction d'ARN pour exprimer une réplicase d'alphavirus comprend une coiffe d'extrémité 5'. Il a été découvert de manière surprenante que la coiffe d'extrémité 5 ' est apte à entraîner efficacement l'expression d'un transgène à partir du réplicon en trans. Le système selon la présente invention permet l'expression d'une protéine d'intérêt dans une cellule ou un organisme, mais n'est pas associé à une formation indésirable de particules virales. Par conséquent, la présente invention est apte à produire de manière efficace et sécurisée une protéine d'intérêt, par exemple une protéine thérapeutique ou une protéine antigénique, telle qu'un vaccin, dans un organisme cible. L'invention concerne des procédés respectifs de production de protéines in vitro et in vivo ainsi que des utilisations médicales. La présente invention concerne également un ADN codant les molécules d'ARN selon l'invention, et des cellules comprenant les molécules d'ARN selon l'invention.
PCT/EP2016/056160 2016-03-21 2016-03-21 Arn à réplication trans WO2017162265A1 (fr)

Priority Applications (31)

Application Number Priority Date Filing Date Title
PCT/EP2016/056160 WO2017162265A1 (fr) 2016-03-21 2016-03-21 Arn à réplication trans
SI201730973T SI3433368T1 (sl) 2016-03-21 2017-03-13 Trans-replikacijska RNA
MDE20190126T MD3433368T2 (ro) 2016-03-21 2017-03-13 Trans-replicarea ARN
MA44479A MA44479B1 (fr) 2016-03-21 2017-03-13 Arn transrepliquant
AU2017236240A AU2017236240B2 (en) 2016-03-21 2017-03-13 Trans-replicating RNA
CA3017285A CA3017285A1 (fr) 2016-03-21 2017-03-13 Arn a replication trans
BR112018068391A BR112018068391A2 (pt) 2016-03-21 2017-03-13 sistema e métodos para produzir uma proteína, célula inoculada, construção de rna para expressar a replicase de alfavírus e dna
HUE17710268A HUE058889T2 (hu) 2016-03-21 2017-03-13 Transz-replikáló RNS
EP21200788.4A EP3964584A1 (fr) 2016-03-21 2017-03-13 Arn transrépliquant
CN202211115420.5A CN115386596A (zh) 2016-03-21 2017-03-13 反式复制rna
US16/086,127 US20200299724A1 (en) 2016-03-21 2017-03-13 Trans-Replicating RNA
PL17710268T PL3433368T3 (pl) 2016-03-21 2017-03-13 Rna z replikacją trans
HRP20211715TT HRP20211715T1 (hr) 2016-03-21 2017-03-13 Trans-replicirajuća rnk
KR1020187027526A KR102441725B1 (ko) 2016-03-21 2017-03-13 트랜스-복제 rna
PCT/EP2017/055813 WO2017162461A1 (fr) 2016-03-21 2017-03-13 Arn à réplication trans
DK17710268.8T DK3433368T3 (da) 2016-03-21 2017-03-13 Transreplikerende rna
PT177102688T PT3433368T (pt) 2016-03-21 2017-03-13 Arn trans-replicante
MX2018011384A MX2018011384A (es) 2016-03-21 2017-03-13 Arn de replicacion en trans.
RS20211377A RS62543B1 (sr) 2016-03-21 2017-03-13 Trans-replicirajuća rnk
JP2018549306A JP7105193B2 (ja) 2016-03-21 2017-03-13 トランス複製型ｒｎａ
ES17710268T ES2896927T3 (es) 2016-03-21 2017-03-13 ARN transreplicante
IL261327A IL261327B1 (en) 2016-03-21 2017-03-13 Trans duplication of RNA
SG11201807378SA SG11201807378SA (en) 2016-03-21 2017-03-13 Trans-replicating rna
EP17710268.8A EP3433368B1 (fr) 2016-03-21 2017-03-13 Arn transrepliquant
CN201780018575.8A CN109328233B (zh) 2016-03-21 2017-03-13 反式复制rna
LTEPPCT/EP2017/055813T LT3433368T (lt) 2016-03-21 2017-03-13 Trans-replikuojama rnr
RU2018131783A RU2752580C2 (ru) 2016-03-21 2017-03-13 Транс-реплицирующая рнк
ZA201805520A ZA201805520B (en) 2016-03-21 2018-08-17 Trans-replicating rna
ZA2019/05396A ZA201905396B (en) 2016-03-21 2019-08-15 Trans-replicating rna
JP2021160491A JP7421256B2 (ja) 2016-03-21 2021-09-30 トランス複製型ｒｎａ
CY20211100949T CY1124689T1 (el) 2016-03-21 2021-11-03 Trans-αντιγραφοymeno rna

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
PCT/EP2016/056160 WO2017162265A1 (fr) 2016-03-21 2016-03-21 Arn à réplication trans

Publications (1)

Publication Number Publication Date
WO2017162265A1 true WO2017162265A1 (fr) 2017-09-28

Family

ID=55586324

Family Applications (2)

Application Number Title Priority Date Filing Date
PCT/EP2016/056160 WO2017162265A1 (fr) 2016-03-21 2016-03-21 Arn à réplication trans
PCT/EP2017/055813 WO2017162461A1 (fr) 2016-03-21 2017-03-13 Arn à réplication trans

Family Applications After (1)

Application Number Title Priority Date Filing Date
PCT/EP2017/055813 WO2017162461A1 (fr) 2016-03-21 2017-03-13 Arn à réplication trans

Country Status (26)

Country Link
US (1) US20200299724A1 (fr)
EP (2) EP3433368B1 (fr)
JP (2) JP7105193B2 (fr)
KR (1) KR102441725B1 (fr)
CN (2) CN109328233B (fr)
AU (1) AU2017236240B2 (fr)
BR (1) BR112018068391A2 (fr)
CA (1) CA3017285A1 (fr)
CY (1) CY1124689T1 (fr)
DK (1) DK3433368T3 (fr)
ES (1) ES2896927T3 (fr)
HR (1) HRP20211715T1 (fr)
HU (1) HUE058889T2 (fr)
IL (1) IL261327B1 (fr)
LT (1) LT3433368T (fr)
MA (1) MA44479B1 (fr)
MD (1) MD3433368T2 (fr)
MX (1) MX2018011384A (fr)
PL (1) PL3433368T3 (fr)
PT (1) PT3433368T (fr)
RS (1) RS62543B1 (fr)
RU (1) RU2752580C2 (fr)
SG (1) SG11201807378SA (fr)
SI (1) SI3433368T1 (fr)
WO (2) WO2017162265A1 (fr)
ZA (2) ZA201805520B (fr)

Cited By (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017015463A3 (fr) * 2015-07-21 2018-04-26 Modernatx, Inc. Vaccins contre une maladie infectieuse
WO2019053056A1 (fr) * 2017-09-13 2019-03-21 Biontech Cell & Gene Therapies Gmbh Réplicon d'arn permettant d'exprimer un récepteur de lymphocyte t ou un récepteur de lymphocyte t artificiel
WO2019053012A1 (fr) * 2017-09-13 2019-03-21 Biontech Rna Pharmaceuticals Gmbh Réplicon d'arn pour la reprogrammation de cellules somatiques
US10653767B2 (en) 2017-09-14 2020-05-19 Modernatx, Inc. Zika virus MRNA vaccines
GB202020063D0 (en) 2020-12-17 2021-02-03 Imperial College Innovations Ltd RNA construct
GB202020064D0 (en) 2020-12-17 2021-02-03 Imperial College Innovations Ltd RNA construct
GB202020061D0 (en) 2020-12-17 2021-02-03 Imp College Innovations Ltd RNA construct
WO2022002783A1 (fr) 2020-06-29 2022-01-06 Glaxosmithkline Biologicals Sa Adjuvants
US11235052B2 (en) 2015-10-22 2022-02-01 Modernatx, Inc. Chikungunya virus RNA vaccines
US11364292B2 (en) 2015-07-21 2022-06-21 Modernatx, Inc. CHIKV RNA vaccines
WO2022200574A1 (fr) 2021-03-26 2022-09-29 Glaxosmithkline Biologicals Sa Compositions immunogènes
WO2022234268A1 (fr) * 2021-05-04 2022-11-10 Mogrify Limited Conversion cellulaire
WO2022248353A1 (fr) 2021-05-24 2022-12-01 Glaxosmithkline Biologicals Sa Adjuvants
US20220396798A1 (en) * 2021-06-12 2022-12-15 Shi-Lung Lin Novel mrna composition and production method for use in anti-viral and anti-cancer vaccines
WO2022264109A1 (fr) 2021-06-18 2022-12-22 Sanofi Vaccins multivalents contre la grippe
WO2023020992A1 (fr) 2021-08-16 2023-02-23 Glaxosmithkline Biologicals Sa Nouveaux procédés
WO2023020994A1 (fr) 2021-08-16 2023-02-23 Glaxosmithkline Biologicals Sa Nouveaux procédés
WO2023021427A1 (fr) 2021-08-16 2023-02-23 Glaxosmithkline Biologicals Sa Lyophilisation de nanoparticules lipidiques (lnp) encapsulant de l'arn et leurs formulations
WO2023021421A1 (fr) 2021-08-16 2023-02-23 Glaxosmithkline Biologicals Sa Vaccins à base d'arn lyophilisés à faible dose et leurs procédés de préparation et d'utilisation
WO2023020993A1 (fr) 2021-08-16 2023-02-23 Glaxosmithkline Biologicals Sa Nouveaux procédés
WO2023066874A1 (fr) 2021-10-18 2023-04-27 BioNTech SE Procédés pour déterminer les mutations permettant d'augmenter la fonction de l'arn modifié réplicable et compositions connexes et leur utilisation
WO2023079507A1 (fr) 2021-11-05 2023-05-11 Sanofi Vaccin à arn contre le virus respiratoire syncytial
WO2023111262A1 (fr) 2021-12-17 2023-06-22 Sanofi Vaccin à base d'arn contre la maladie de lyme
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WO2023214082A2 (fr) 2022-05-06 2023-11-09 Sanofi Séquences de signaux pour vaccins à base d'acides nucléiques
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